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Synonyms: BIIB-067 | BIIB067 | Qalsody®
tofersen is an approved drug (FDA (2023), EMA (2024))
Compound class:
Nucleic acid
Comment: Tofersen (BIIB067) is an antisense oligonucleotide that disrupts translation of superoxide dismutase 1 (SOD1). Tofersen was designed to treat cases of amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) that are associated with expression of mutated SOD1. SOD1-ALS/MND accounts for ~2% of all cases.
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Nucleic Acid Sequence  |
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| (3'-5)-mC=A-G=G-A=dT=dA=dmC=dA=dT=dT=dT=dmC=dT=dA=mC-A=G-mC=mU | |
